Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Fibrocell Science and Intrexon are planning to submit an investigational new drug (IND) application with the US Food and Drug Administration (FDA) in the first half of 2015 for their drug candidate, GM-HDF-COL7 (genetically-modified human dermal fibroblast, collagen VII) to treat recessive dystrophic epidermolysis bullosa (RDEB).
Subscribe to our email newsletter
RDEB is the most severe form of the rare connective tissue disorder epidermolysis bullosa and it causes severe blistering and areas of missing skin that occurs when a mutation or error happens within the collagen VII gene.
Earlier in 2014, the companies started preclinical activities of GM-HDF-COL7.
The companies have also secured certain regulatory and development achievements including orphan drug designation in June, a collaborative pre-IND meeting with the FDA in August, and start of clinical material manufacturing in September.
Intrexon Health Sector senior vice-president Gregory Frost said the company is happy with the progress of its collective efforts with Fibrocell to bring this new class of engineered therapeutics closer to the clinic to address the medical needs of patients with this rare and devastating disease.
"Correcting a gene as large as the one disrupted in these patients through a genetically-modified cellular therapy highlights the potential of the UltraVector platform to deliver solutions for diseases previously deemed untreatable," Frost said.
Fibrocell chairman and chief executive officer David Pernock said: "Today during our R&D Day, a more complete picture of Fibrocell’s growth strategy will emerge, and we will present a deeper dive into the GM-HDF-COL7 program for RDEB, as well as a range of programs that deploy our unique autologous fibroblast technology."